electronic health records – Clinical Research Made Simple

Integrating Real-World Evidence in Rare Disease Clinical Trials

August 23, 2025

Integrating Real-World Evidence in Rare Disease Clinical Trials Harnessing Real-World Evidence for Rare Disease Clinical Trial Success Why Real-World Evidence Matters in Rare Disease Studies Rare disease trials often face unique challenges—small sample sizes, heterogeneous patient populations, and ethical concerns with placebo use. Real-world evidence (RWE), derived from electronic health records (EHRs), patient registries, insurance…

Data & Technology, Rare and Orphan Disease Trials

Real-World Data Impact on Rare Disease Drug Label Expansion

August 15, 2025

digi

Real-World Data Impact on Rare Disease Drug Label Expansion How Real-World Data Is Driving Drug Label Expansion in Rare Diseases Introduction: Why Real-World Data Matters in Rare Diseases Rare disease clinical development is often limited by small patient populations, short trial durations, and narrowly defined eligibility criteria. This can result in regulatory approvals that are…

Case Studies & Breakthroughs, Rare and Orphan Disease Trials